30 September 2019
New Zealand’s first clinical trial of CAR T-cell therapy, a revolutionary new approach to fighting cancer, is getting underway after receiving final regulatory approval.
The phase I safety trial, called ENABLE, is part the of the Malaghan Institute’s research and development of a new version of CAR T-cell technology, in partnership with Wellington Zhaotai Therapies Limited. The trial will involve up to 12 participants with certain types of relapsed and refractory B-cell non-Hodgkin lymphoma who have exhausted other treatment options.
Malaghan Institute Clinical Director Dr Robert Weinkove says the production of CAR T-cells is a major step in the development of the Institute’s cell therapy capabilities.
“Chimeric antigen receptor T-cell therapies are being offered to treat certain types of B-cell lymphoma (lymph node cancer) in countries such as Australia and the UK. For this early phase safety trial of a new type of CAR T-cell therapy, we’ll be manufacturing the cells in the dedicated cell therapy suite at the Malaghan Institute in Wellington.
“Because the safety and effectiveness are not yet known, this will be a small trial for a limited number of participants. Nonetheless, this is a very exciting milestone, and we hope the experience and knowledge we gain from the ENABLE trial will help more New Zealanders benefit from CAR T-cell therapies in the future.”
Dr Weinkove says the trial will not be the right option for everyone, and it is important that patients speak with their specialist about their treatment options. “Referrals for the trial can only be accepted from haematologists or oncologists, who will be advised of the criteria and how to refer.”
Dr Weinkove says the trial is taking place at a single centre, to allow close monitoring of participants, but referrals from other hospitals will be considered. “With support from Leukaemia & Blood Cancer New Zealand, we hope to be able to provide travel and accommodation support for participants from outside our region.”
Phase I trials assess the safety of a new treatment, and determine the optimal dose. Participants will be recruited gradually, to allow safety monitoring between each participant. Dr Weinkove says it is unclear exactly when the first participant will receive treatment, as this will depend on the results of pre-treatment tests, successful CAR T-cell manufacture and on ensuring that all necessary checks are satisfied before the cell are given.
“We anticipate that it will take 18 months to complete recruitment to this trial. While we will get preliminary response data over the course of the trial, full analysis of the primary outcome data could take up to a further year.”
CAR T-cell therapy is an emerging form of cancer treatment which has recently been licensed in Australia, the United States and Europe to treat patients with certain types of lymphoma and leukaemia. The Malaghan Institute’s CAR T-cell product is a new ‘third generation’ CAR T-cell technology, based on new and unique intellectual property.
“While we hope our CAR T-cells’ unique properties translate to clinical benefits, they may not. The differences mean we cannot rely on results of trials undertaken with other CAR T-cell products overseas, and must establish the optimal dose and safety of our cells before we can progress to larger trials to establish its effectiveness.”
Dr Weinkove says the aim of the Institute’s Freemasons CAR T-cell Research Programme and its clinical programme is to accelerate availability of the treatment locally.
“By establishing the production of CAR T-cells for research and early phase clinical trials, we hope to develop and support the regulatory and clinical environment for safe CAR T-cell delivery in New Zealand.
“We are also undertaking parallel laboratory research focusing on improving CAR T-cell therapies and potentially extending them to other cancers in future,” says Dr Weinkove.
“This is an exciting time for cancer research, only made possible thanks to the support of a whole cast of donors and funders including Freemasons New Zealand, David Downs’ Down with Cancer campaign, the Thompson Family Foundation, the Florence Petersen Leukaemia Trust, the Hugh Green Foundation, the K.I.A. Taylor Charitable Trust, the Tonks Family Foundation, the Lion Foundation, the Infinity Foundation, the Health Research Council and the Ministry for Business, Innovation and Employment.”
The Malaghan Institute is not a provider of health services and does not recruit patients to clinical trials directly. Referrals to the CAR T-cell ENABLE trial must be made by a relevant specialist (a haemotologist or oncologist) to the Clinical Trials Unit via RES-Research@ccdhb.org.nz or www.ccdhb.org.nz/working-with-us/clinical-trials-unit/